Cure SMA Announces Nearly $2 Million in New Planned Research Funding

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Cure SMA has announced plans to fund $1.995 million in new research grants over the next several months. This includes funding for all four areas of our research model—basic research, drug discovery, clinical trials, and clinical care research—plus funding for the 2016 SMA Researcher Meeting, held each year as part of the Annual SMA Conference. […]

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Dr. Mary Schroth Joins Cure SMA as Medical Professional Education Consultant

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We are very excited to announce that Dr. Mary Schroth has joined us as our medical professional education consultant. She will be working with us a few days each month, depending on her clinic schedule, as Mary remains deeply committed to working with her patients. Mary will also continue to chair our Medical Advisory Council.

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The 2015 Annual SMA Conference is Here!

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The 26th Annual SMA Conference kicks off tomorrow with our Newly Diagnosed Program, and continues into the weekend with all of our usual programs that bring families, researchers, and healthcare providers together to network, learn and —plus a few new initiatives. This will be one of our largest conferences ever. We’re expecting over 1,300 attendees

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2015 Annual SMA Conference Exhibitors

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Thank you to the following exhibitors who have generously supported our 2015 Annual SMA Conference. This one-of-a-kind event would not be possible without your support! For more information on any of these companies, please visit our conference exhibitor page. Platinum Ki MobilityQuantum Rehab Gold 3E Love Bio-Rad Laboratories, Inc. Convaid Neotech Products Percussionaire Permobil PromptCare

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Cure SMA Releases New Clinical Trial Care Series Booklet

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As more and more SMA drug programs progress through clinical trials, there is an increasing need for accurate and unbiased information on clinical trials from our community. Cure SMA has been working to address this through education efforts targeted to the specific needs our community. The centerpiece of these education efforts is our new care

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Isis Releases Encouraging Results from Phase 2 Study

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Isis Pharmaceuticals provided an update on its ongoing open-label Phase 2 clinical study of ISIS-SMNRx in infants with Type I spinal muscular atrophy (SMA). Previously the company reported data from this study on event-free survival, measures of muscle function and assessments of developmental milestones. The data reported now shows continued increases in median event-free survival

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SMA Drug Pipeline Grows to 18 Programs

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We’ve recently released an update to our SMA drug pipeline. This latest version shows the continued growth of the pipeline, setting several new marks for both breadth and depth: We have 18 active programs. We have 15 pharmaceutical partners. We have seven programs in clinical trials. The cumulative pipeline total is 26 programs, including eight

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Spring 2015 Directions Posted Online

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The Spring 2015 issue of Directions, our biannual family support newsletter, builds our community by sharing stories and experiences. Families from all over the world connect with each other through Cure SMA to chat, organize, and share stories, photos, news, information, and—most importantly—hope. This issue includes information on the upcoming 2015 Annual SMA Conference, news

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Biogen Announces EMBRACE, a New Phase 2 Clinical Trial

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Biogen, in partnership with Isis Pharmaceuticals, has announced a new Phase 2 clinical trial, called EMBRACE. EMBRACE is a multi-center, Phase 2 clinical study evaluating the safety and exploratory efficacy of the investigational drug, ISIS-SMNRx, (ISIS 396443) in patients with infantile or childhood-onset Spinal Muscular Atrophy (SMA). The study is a double-blind, randomized, sham-procedure controlled

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